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Efficient Ablation ...
Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9.
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Mandal, Pankaj K (author)
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Ferreira, Leonardo M R (author)
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Collins, Ryan (author)
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Meissner, Torsten B (author)
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Boutwell, Christian L (author)
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Friesen, Max (author)
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Vrbanac, Vladimir (author)
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Garrison, Brian S (author)
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Stortchevoi, Alexei (author)
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- Bryder, David (author)
- Lund University,Lunds universitet,Institutionen för experimentell medicinsk vetenskap,Medicinska fakulteten,Department of Experimental Medical Science,Faculty of Medicine
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Musunuru, Kiran (author)
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Brand, Harrison (author)
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Tager, Andrew M (author)
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Allen, Todd M (author)
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Talkowski, Michael E (author)
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Rossi, Derrick J (author)
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Cowan, Chad A (author)
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(creator_code:org_t)
- Elsevier BV, 2014
- 2014
- English.
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In: Cell Stem Cell. - : Elsevier BV. - 1934-5909. ; 15:5, s. 643-652
- Related links:
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http://www.ncbi.nlm....
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http://dx.doi.org/10...
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http://www.cell.com/...
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
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- Genome editing via CRISPR/Cas9 has rapidly become the tool of choice by virtue of its efficacy and ease of use. However, CRISPR/Cas9-mediated genome editing in clinically relevant human somatic cells remains untested. Here, we report CRISPR/Cas9 targeting of two clinically relevant genes, B2M and CCR5, in primary human CD4(+) T cells and CD34(+) hematopoietic stem and progenitor cells (HSPCs). Use of single RNA guides led to highly efficient mutagenesis in HSPCs but not in T cells. A dual guide approach improved gene deletion efficacy in both cell types. HSPCs that had undergone genome editing with CRISPR/Cas9 retained multilineage potential. We examined predicted on- and off-target mutations via target capture sequencing in HSPCs and observed low levels of off-target mutagenesis at only one site. These results demonstrate that CRISPR/Cas9 can efficiently ablate genes in HSPCs with minimal off-target mutagenesis, which could have broad applicability for hematopoietic cell-based therapy.
Subject headings
- NATURVETENSKAP -- Biologi -- Cellbiologi (hsv//swe)
- NATURAL SCIENCES -- Biological Sciences -- Cell Biology (hsv//eng)
Publication and Content Type
- art (subject category)
- ref (subject category)
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- By the author/editor
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Mandal, Pankaj K
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Ferreira, Leonar ...
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Collins, Ryan
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Meissner, Torste ...
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Boutwell, Christ ...
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Friesen, Max
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show more...
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Vrbanac, Vladimi ...
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Garrison, Brian ...
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Stortchevoi, Ale ...
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Bryder, David
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Musunuru, Kiran
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Brand, Harrison
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Tager, Andrew M
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Allen, Todd M
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Talkowski, Micha ...
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Rossi, Derrick J
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Cowan, Chad A
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show less...
- About the subject
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- NATURAL SCIENCES
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NATURAL SCIENCES
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and Biological Scien ...
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and Cell Biology
- Articles in the publication
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Cell Stem Cell
- By the university
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Lund University